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RCKT

Rocket Pharmaceuticals

$16.00

0.23 (1.46%)

07:03
01/22/19
01/22
07:03
01/22/19
07:03

Rocket Pharmaceuticals announces FDA clearance of IND application for RP-A501

Rocket Pharmaceuticals announces the clearance of the Company's Investigational New Drug application by the U.S. Food and Drug Administration for RP-A501. RP-A501 is the Company's first adeno-associated viral vector-based gene therapy for the treatment of Danon disease that is in development under a collaboration with the University of California San Diego School of Medicine. UC San Diego Health will be the initial and lead center for the planned Phase 1 clinical trial under the direction of Eric Adler, M.D., Director of Cardiac Transplant and Mechanical Circulatory Support at UC San Diego Health and Professor of Medicine at University of California San Diego School of Medicine. In parallel with the Phase 1 clinical trial, the Company plans to publish a comprehensive literature review of Danon cases and conduct a retrospective chart review. In addition, the Company will initiate a prospective natural history study with enrollment planned to commence in the first quarter of 2019.

RCKT Rocket Pharmaceuticals
$16.00

0.23 (1.46%)

07/10/18
WBLR
07/10/18
INITIATION
WBLR
Outperform
William Blair starts Rocket Pharmaceuticals with Outperform, $32 fair value
William Blair analyst Raju Prasad initiated coverage of Rocket Pharmaceuticals with an Outperform rating and $32 fair value estimate. The company is focused on developing first-in-class gene therapies for rare diseases, Prasad tells investors in a research note. The company's lead candidate, RP-L102, is an ex vivo lentiviral gene therapy being developed for the treatment of Fanconi anemia, with three additional ex vivo lentivirus programs in earlier development, the analyst adds. He believes leukocyte adhesion deficiency-I and pyruvate kinase deficiency could generate about $530M and $420M in revenue by 2027, respectively.
09/13/18
LTCO
09/13/18
INITIATION
Target $30
LTCO
Buy
Rocket Pharmaceuticals initiated with a Buy at Ladenburg
Ladenburg Thalmann analyst Jonathan Hickman started Rocket Pharmaceuticals with a Buy rating and $30 price target. The analyst sees the company's "large" pipeline fueling its "rising" gene therapy portfolio.
11/27/18
LEER
11/27/18
INITIATION
Target $22
LEER
Outperform
Rocket Pharmaceuticals initiated with an Outperform at Leerink
Leerink analyst Mani Foroohar initiated Rocket Pharmaceuticals with an Outperform rating and a price target of $22.
11/27/18
LEER
11/27/18
INITIATION
LEER
Leerink initiates select Genetic Medicine names on increasing regulatory clarity
Leerink analyst Mani Foroohar initiated select Genetic Medicine stocks, stating that the field is "is rich with companies developing drugs" to treat rare diseases and that he is increasingly more bullish on "innovation velocity and increasing regulatory clarity in the sector. Based on his "balanced view of pricing dynamics", the analyst issues an Outperform rating on Wave Life Sciences (WVE), Rocket Pharmaceuticals (RCKT), Intellia Therapeutics (NTLA), Eidos Therapeutics (EIDX), Dicerna (DRNA) and Bluebird Bio (BLUE). The analyst likes Wave Life Sciences as a pure play on Oligotherapeutics and its stereopure chemistry platform as a differentiated approach to oligo drug design. Foroohar is positive on Rocket Pharma's lead assets in gene therapies for Fanconi Anemia and Leukocyte Adhesion Deficiency-I "which will likely be the company's first potential approved product." The analyst is positive on Intellia Therapeutics' "robust balance sheet to support development, trading at a material discount to closest comps peers". For Eidos, the analyst cites the prospects of its drug candidate for TTR amyloidosis currently in a placebo-controlled Phase 2 study that may offer a competitive profile relative to Pfizer's (PFE) tafamidis. For Dicerna, Foroohar notes that its DCR-PHXC "is an RNAi therapeutic that targets lactate dehydrogenase A for the treatment of all subtypes of Primary Hyperoxaluria vs. competitor Alnylam's (ALNY) lumasiran, which only treats PH Type 1. For Bluebird Bio, the analyst is positive on its position as a "leader in ex vivo gene therapy, with four programs with visibility towards near-term regulatory approval". Foroohar initiates Avrobio (AVRO), Alnylam (ALNY), and Ionis Pharmaceuticals (IONS) with Market Perform rating based on competitive launches for their products along with "mixed" early signs of their results. The analyst also rates Regenxbio (RGNX) at underperform, saying that its "current valuation more than accounts for robust royalty streams from sales in spinal muscular atrophy" and other partnered assets.

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